EU/3/15/1442 - orphan designation for treatment of Ebola virus disease

fibrinogen-coated albumin spheres

Overview

On 12 February 2015, orphan designation (EU/3/15/1442) was granted by the European Commission to Fibreu Limited, United Kingdom, for fibrinogen-coated albumin spheres for the treatment of Ebola virus disease.

The sponsorship was transferred to Fibreu Limitless Research S.L., Spain, in May 2019.

What is Ebola virus disease?

Ebola virus disease is a severe disease caused by infection with viruses known as ebolaviruses. There are 5 known species of ebolavirus, 4 of which are known to cause the disease in humans. Zaire ebolavirus, sometimes referred to simply as 'ebola virus' or EBOV, is the cause of the largest outbreaks of the disease to date and has led to the most deaths.

Infection is caused by contact with the body fluids of an infected person. After infection there is an incubation period of between 2 to 21 days, following which the newly infected person starts to experience symptoms. The first symptoms typically are fever, headache, fatigue, muscle pain and sore throat. These are followed by other symptoms such as diarrhoea, vomiting, rash, kidney and liver problems and, in some cases, internal bleeding and bleeding from the gums, eyes, nose and ears. Patients are infectious once they start experiencing symptoms.

Ebola virus disease is a life-threatening condition that is frequently fatal due to fluid loss and severe bleeding.

What is the estimated number of patients affected by the condition?

At the time of designation, Ebola virus disease affected less than 0.01 in 10,000 people in the European Union (EU). This is below the ceiling for orphan designation, which is 5 people in 10,000. This is based on the information provided by the sponsor and the knowledge of the Committee for Orphan Medicinal Products (COMP).

What treatments are available?

At the time of application for orphan designation, there were no satisfactory treatments authorised in the EU for treating Ebola virus disease.

How is this medicine expected to work?

Patients with Ebola virus disease can suffer from severe life-threatening bleeding, especially when the disease leads to low levels of blood platelets, cells that play a role in blood clotting. This medicine consists of spherical particles coated with fibrinogen, a protein naturally found in the blood that is also involved in helping the blood to clot. When the particles reach a bleeding site, the extra fibrinogen in the medicine is converted into a fibrous protein known as fibrin, which helps plug the bleeding site, in the same way as a natural blood clot. This additional clotting effect is expected to help counteract any clotting problems caused by lack of blood platelets and so to reduce the bleeding associated with the disease.

What is the stage of development of this medicine?

The effects of the medicine have been evaluated in experimental models.

At the time of submission of the application for orphan designation, no clinical trials with the medicine in patients with Ebola virus disease had been started.

At the time of submission, the medicine was not authorised anywhere in the EU for Ebola virus disease or designated as an orphan medicinal product elsewhere for this condition.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 9 January 2015 recommending the granting of this designation.

Opinions on orphan medicinal product designations are based on the following three criteria:

• the seriousness of the condition;
• the existence of alternative methods of diagnosis, prevention or treatment;
• either the rarity of the condition (affecting not more than 5 in 10,000 people in the EU) or insufficient returns on investment.

Designated orphan medicinal products are products that are still under investigation and are considered for orphan designation on the basis of potential activity. An orphan designation is not a marketing authorisation. As a consequence, demonstration of quality, safety and efficacy is necessary before a product can be granted a marketing authorisation.

EU/3/15/1442: Public summary of opinion on orphan designation: Fibrinogen-coated albumin spheres for the treatment of Ebola virus disease

Adopted Reference Number: EMA/COMP/55779/2015

Key facts

• Active substance:
  fibrinogen-coated albumin spheres
• Intended use:
  Treatment of Ebola virus disease
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/15/1442
• Date of designation:
  12/02/2015
• Sponsor:

  Fibreu Limitless Research S.L.; Calle Lopez De Neira Num. 3; Planta 3 Puerta 301; Vigo; Pontevedra 36202; Spain; Tel. [phone]; E-mail: [e-mail]

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.